Development of iPSC-Derived Myocyte Models with PABPN1 Repeat Expansions for Antisense Oligonucleotide Therapy in Oculopharyngeal Muscular Dystrophy (OPMD)

Description

This project aims to develop an innovative cellular model of oculopharyngeal muscular dystrophy (OPMD) using myocytes derived from induced pluripotent stem cells (iPSC) modified by CRISPR to reproduce trinucleotide repeat expansions in the PABPN1 gene. The student will test the efficacy of antisense oligonucleotide (ASO) therapy to reduce toxic PABPN1 protein aggregates and restore muscle function, drawing on the CHU de Québec clinical cohorts and contributing to the preclinical validation of translational treatments for this rare progressive disease.

Research Field

- Oculopharyngeal muscular dystrophy (OPMD)
- Induced pluripotent stem cells (iPSC)
- CRISPR genome editing
- Antisense oligonucleotides (ASO)
- PABPN1 protein
- Toxic protein aggregates
- 3D cellular modeling
- Translational gene therapy

Research Supervisor

Vincent Picher-Martel

Research Environment

CHU de Québec - Université Laval research Center

The Centre de recherche du CHU de Québec-Université Laval is a highly collaborative research environment, particularly conducive to neuroscience and neuromuscular disorders through its Neurosciences axis, which brings together experts in basic and clinical research. This axis focuses on understanding nervous and neuromuscular systems in physiological and pathological conditions, with key themes such as cellular and molecular neurobiology, relevant to Dr. Picher-Martel's projects on ALS, oculopharyngeal muscular dystrophy, induced pluripotent stem cells, and tissue modeling. Teams benefit from state-of-the-art infrastructure (multimodal imaging, genomics, proteomics, electrophysiology) and foster national/international multicenter collaborations for rapid translational studies toward clinical care. This dynamic environment supports innovation in neurology, training of the next generation, and the transfer of discoveries into therapies for neuromuscular diseases.)

Web Site

Financial Aid Available by Program of Study

Doctorate in Cellular and Molecular Biology

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
Program progress award (S-1 to S-8)	8 x $525
Program progress award (S-9 to S-11)	3 x $200
Successful completion of Ph.D exam	$800
Submission of dissertation deposit before the end of the 12th semester	$2,000
Language training	$250
Total	$7,850

Université Laval: Student Financial Aid

Funding source	Amount
Leadership and Comittment Scholarship (Canadians & permanent residents)	$30,000
Citizens of the World Scholarship (International students)	$30,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$25,000 to $40,000$
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

Supplemental Tuition Fee Exemption Scholarship Program: Entitles international students to pay Canadian student tuition fees, for overall savings of around $45,000.

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Doctorate in Clinical and Biomedical Sciences

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
Program progress award (S-1 to S-8)	8 x $525
Program progress award (S-9 to S-11)	3 x $200
Successful completion of Ph.D exam	$800
Submission of dissertation deposit before the end of the 12th semester	$2,000
Language training	$250
Total	$7,850

Université Laval: Student Financial Aid

Funding source	Amount
Leadership and Comittment Scholarship (Canadians & permanent residents)	$30,000
Citizens of the World Scholarship (International students)	$30,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$25,000 to $40,000$
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

Supplemental Tuition Fee Exemption Scholarship Program: Entitles international students to pay Canadian student tuition fees, for overall savings of around $45,000.

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Doctorate in Molecular Medicine

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
Program progress award (S-1 to S-8)	8 x $525
Program progress award (S-9 to S-11)	3 x $200
Successful completion of Ph.D exam	$800
Submission of dissertation deposit before the end of the 12th semester	$2,000
Language training	$250
Total	$7,850

Université Laval: Student Financial Aid

Funding source	Amount
Leadership and Comittment Scholarship (Canadians & permanent residents)	$30,000
Citizens of the World Scholarship (International students)	$30,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$25,000 to $40,000$
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

Supplemental Tuition Fee Exemption Scholarship Program: Entitles international students to pay Canadian student tuition fees, for overall savings of around $45,000.

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Doctorate in Neurosciences

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
Program progress award (S-1 to S-8)	8 x $525
Program progress award (S-9 to S-11)	3 x $200
Successful completion of Ph.D exam	$800
Submission of dissertation deposit before the end of the 12th semester	$2,000
Language training	$250
Total	$7,850

Université Laval: Student Financial Aid

Funding source	Amount
Leadership and Comittment Scholarship (Canadians & permanent residents)	$30,000
Citizens of the World Scholarship (International students)	$30,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$25,000 to $40,000$
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

Supplemental Tuition Fee Exemption Scholarship Program: Entitles international students to pay Canadian student tuition fees, for overall savings of around $45,000.

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Master's Degree in Cellular and Molecular Biology with thesis

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
First submission of thesis by the end of the 5th semester of full-time registration	$2,000
Language training	$250
Total	$2,250

Université Laval: Student Financial Aid

Sources de financement	Montant
Leadership and Comittment Scholarship (Canadians & permanent residents)	$10,000
Citizens of the World Scholarship (International students)	$20,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$20,000 to $27,000
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Master's Degree in Clinical and Biomedical Sciences with thesis

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
First submission of thesis by the end of the 5th semester of full-time registration	$2,000
Language training	$250
Total	$2,250

Université Laval: Student Financial Aid

Sources de financement	Montant
Leadership and Comittment Scholarship (Canadians & permanent residents)	$10,000
Citizens of the World Scholarship (International students)	$20,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$20,000 to $27,000
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Master's Degree in Molecular Medicine with thesis

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
First submission of thesis by the end of the 5th semester of full-time registration	$2,000
Language training	$250
Total	$2,250

Université Laval: Student Financial Aid

Sources de financement	Montant
Leadership and Comittment Scholarship (Canadians & permanent residents)	$10,000
Citizens of the World Scholarship (International students)	$20,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$20,000 to $27,000
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Master's Degree in Neurosciences with thesis

Program description

Financial Aid Available*

Financial Aid Related to Research Project

Information unavailable

Program-Specific Financial Aid

Graduate Studies Awards

Milestone	Amount
First submission of thesis by the end of the 5th semester of full-time registration	$2,000
Language training	$250
Total	$2,250

Université Laval: Student Financial Aid

Sources de financement	Montant
Leadership and Comittment Scholarship (Canadians & permanent residents)	$10,000
Citizens of the World Scholarship (International students)	$20,000
Mobility Grant for Out-of-Province Internships or Research Visits (in French)	$1,500 to $3,000
Graduate Scholarships from Granting Agencies	$20,000 to $27,000
Online Directory of Graduate Scholarships (in French)	$500 to $50,000

_{* Amounts shown represent maximum financial aid available. Certain conditions apply. Subject to change without prior notice. For further information, contact sponsoring organizations directly.}

Desired Profile

- Medecine
- Occupational Therapy
- Speech Therapy
- Physiotherapy
- Biomedical Science
- Neurology
- Biochemistry
- Biology
- Endocrinology and Metabolism
- Neuropathology
- Microbiology

Required Documentation

- Cover letter
- Curriculum vitæ
- Student transcript

Find Out More

Mathieu Blais
Dr - Chargé de projets
Centre de recherche du CHU de Québec
mathieu.blais@crchudequebec.ulaval.ca